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EBMT 2020 Annual Meeting - Van Bekkum Award

Severe Aplastic Anaemia Working Party (SAAWP)

Presidential Symposium – Monday 31 August, 14:30 – 16:00H, Auditorium 1

This year’s van Bekkum award has been awarded jointly to Régis Peffault de Latour, Carlo Dufour and Antonio Risitano for their study: “Results of the EBMT Severe Aplastic Anemia Working Party (SAAWP) phase 3 prospective randomised multicentre study of horse ATG and ciclosporin with or without eltrombopag in naïve SAA patients”

The winning abstract can be viewed here:

Q: Congratulations on receiving this prestigious award. For delegates that do not know you, please can you tell us a little about yourselves?

A: Régis: 44 years old, working in Saint-Louis Hospital, in Paris in the BMT unit. Also head of the French reference center for aplastic anaemia and PNH. Current chair of the SAAWP of the EBMT.

Carlo: 63 years old, chairman of the Hem-Onco-SCT Department of the G.Gaslini Children Hospital, Genova, Italy. Also chairman of the Granulocyte and Constitutional Marrow Failure Scientific Working Group of the European Hematology Association (EHA).

Antonio: 46 years old, director of BMT program at the Federico II University of Naples, reference center for aplastic anaemia and paroxysmal nocturnal hemoglobinuria.

Q: What is the background to your study, where did the idea come from and how did you get it started?

A: Aplastic anaemia (AA) is a small club worldwide, and investigators are used to sharing preliminary data and experiences as soon as they are available. The initial results with eltrombopag in refractory AA patients triggered a growing interest to investigate eltrombopag in combination with immunosuppression, and in earlier phases of disease.

While the US National Institutes of Health (NIH) started with a pivotal phase II study treating naïve SAA patients with ATG+CSA+eltrombopag first line (who eventually showed excellent results), we thought it was best to run the phase 3 trial in Europe relying on a higher level of evidence: a prospective randomised controlled trial (RCT). We thus decided to approach GSK who owned eltrombopag at that time, as well as Pfizer to provide horse ATG since ATGAM was not approved on the market at that time (and is still not). Alexion was also part of the discussion. We also approached EBMT as we thought they would be the best sponsor for this kind of study – they agreed, and sponsored the study with the financial support of Novartis and Pfizer. 

Q: What were the main findings of the study and how will they be applied in clinical practice?

Severe aplastic anaemia (SAA) is a rare disease, still potentially lethal, that can be treated with immunosuppression. The most commonly used immunosuppressive regimen includes horse ATG (hATG) in combination with ciclosporine A (CsA). In the RACE trial two arms were compared, with patients randomly treated either in the standard arm including hATG plus CsA or in the arm in which eltrombopag was added to these two medications.

We were able to show that, after three months of treatment, patients who received the combination of hATG, CsA plus eltrombopag had a significantly higher complete response rate, which was also the case at 6 months. In this randomised controlled trial, that represents the highest available of evidence, we thus show that in naïve patients with SAA the addition of eltrombopag to hATG and CsA significantly increases the response rate over the use of hATG and CsA alone without causing any more severe adverse events.

Long-term follow up data will eventually document the impact of this treatment strategy on long-term overall survival and disease-free survival. Based on available data and on the well-known association between haematological response and long-term outcome, the triple therapy with eltrombopag, hATG and CsA will likely become the reference treatment for naïve patients with SAA not eligible for transplantation.

Q: Tell us how you felt on hearing you had been awarded the van Bekkum award

Régis: I would say I feel very proud of all what has been achieved since RACE and my first thoughts were to acknowledge Carlo and Antonio, the Clinical Trial Office (CTO) of the EBMT, together with all RACE investigators and nurses for their outstanding work. I also quickly realised that work still needs to be done to allow patients across Europe to have access to this treatment. EMA recently refused to approve eltrombopag in this indication. These results will certainly help our patients to receive the best care, but the road ahead is still very long!

Carlo: I also felt very proud and honoured to receive this award. I consider the van Bekkum award a prestigious recognition for the extremely valuable teamwork we put in place with my friends Regis and Antonio, with the CTO and the statisticians of the EBMT and with colleagues from all centres that participated to the RACE trial. I also felt very happy for the patients with SAA since the results from RACE will surely generate benefits for them.

Antonio: I felt very honoured and proud to be appointed with such a prestigious award – for sure the most desired for a European transplantologist! This is a great recognition for the wonderful team who did tremendous work (Regis and Carlo, the CTO of the EBMT, together with all RACE investigators and nurses), clearly demonstrating that the “EBMT spirit” pioneered in the 1970s is still alive and inspiring academic research. Experiences like the RACE trials represent the best acknowledgement to the early masters of aplastic anaemia who introduced me to the field, as well as to all patients and families that hopefully will benefit from these therapeutic advances.